Treatment of genetic neuromuscular diseases
by using its tricyclo-DNA molecular technology

ABOUTUS

Synthena’s mission is to develop first in class drugs in the field of Myopathy with a focus on the Duchenne Muscular Dystrophy disease, based on its proprietary tricyclo-DNA technology.

Its proprietary tricyclo-DNA technology offers broad advantages over state of the art oligonucleotide chemistries for new RNA intervention strategies like splice switching, and other antisense approaches. Its most advanced preclinical drug development program focuses on Duchenne Muscular Dystrophy, a genetic disorder which affects 1 in 3,500 boys and which leads to death in early adulthood.

Modulation of RNA for therapeutic purposes holds the promise of opening up entirely new concepts for pharmaceutical intervention, however, current oligonucleotide chemistries to modulate RNA do not meet the requirements for efficient delivery, pharmacokinetics and tolerability.

Leadership

Management Team

Directors & Founders

Management Team

BertrandDamour

Chief Executive Officer

Mr. Damour has been a Biotech Executive for more than 15 years. Most recently he was Chief Executive Officer at OncoEthix a Swiss oncology company sold to Merck & Co. Before that Mr. Damour was Chief Executive Officer of Mind NRG a Swiss company in the field of major CNS pathologies sold to Minerva Neurosciences. Prior to this, Mr. Damour was Executive Vice President and Chief Financial Officer at Egalet, a Danish specialty pharmaceuticals company, now listed on the Nasdaq. Previously, Mr. Damour

was President and Chief Executive Officer at Geneprot, a US biotechnology company in the field of Proteomics and Genomics. Before joining the biotech industry, Mr. Damour was an investment banker holding senior positions at Bankers Trust in New York and at Deutsche Bank, Rabobank and JP Morgan in London. Mr. Damour received his M.B.A. from the Kellogg School of Management at Northwestern University, a Master of Engineering degree from the Institut des Sciences de la Matiere et du Rayonnement (ISMRA) in France, and a Master in Accounting and Business Administration degree from the Institut d’Aministration des Entreprises (IAE) in France. less

BranislavDugovic

PhD, Chief Technology Officer

Studied chemistry at the Slovak technical university since 2005. After performing post-doctoral work at the Freie Universität Berlin he moved to University of Bern in 2009, where he further developed and expanded the bicyclo- and tricyclo-DNA molecular platforms. He joined Synthena in 2012.

AdrianHäberli

PhD, Head Quality Control

Studied chemistry at the University of Bern and graduated 2001 in the subject of oligonucleotide chemistry. Following his postdoctoral studies in Canada he held positions at Interlabor and ZLB Behring. In 2005 he became head of the laboratory unit Pharmacopoeia and scientific expert for forensic chemistry at the Swiss Medicines Control Agency Swissmedic.

Directors & Founders

Bertrand Damour

Chairman of the Board

Mr. Damour has been a Biotech Executive for more than 15 years. Most recently he was Chief Executive Officer at OncoEthix a Swiss oncology company sold to Merck & Co. Before that Mr. Damour was Chief Executive Officer of Mind NRG a Swiss company in the field of major CNS pathologies sold to Minerva Neurosciences. Prior to this, Mr. Damour was Executive Vice President and Chief Financial Officer at Egalet, a Danish specialty pharmaceuticals company, now listed on the Nasdaq. Previously, Mr. Damour

was President and Chief Executive Officer at Geneprot, a US biotechnology company in the field of Proteomics and Genomics. Before joining the biotech industry, Mr. Damour was an investment banker holding senior positions at Bankers Trust in New York and at Deutsche Bank, Rabobank and JP Morgan in London. Mr. Damour received his M.B.A. from the Kellogg School of Management at Northwestern University, a Master of Engineering degree from the Institut des Sciences de la Matiere et du Rayonnement (ISMRA) in France, and a Master in Accounting and Business Administration degree from the Institut d’Aministration des Entreprises (IAE) in France. less

Christian Leumann

PhD

Serves as president of the University of Bern since August 2016. He holds a PhD in chemistry since 1986. After postdoctoral work at the University of Berkeley he returned to ETH Zurich for persuing an academic career. He was appointed professor of bioorganic chemistry at the University of Bern in 1993. Over the last 15 years he developed amongst other projects the tricyclo-DNA molecular platform.

Luc Pettavino

Is directing the Association Monégasque contre les Myopathies (AMM). AMM is heavily involved in supporting research towards cures for Duchenne muscular dystrophy by financing and coordinating the international initiative ICE (International Collaborative Effort) and the Monaco Round Tables since 2003. Prior to this position, Luc Pettavino was CEO and shareholder of the Monaco Yacht Show. He created and organizes the biennal charity auction ONLY WATCH.

Luis Garcia

PhD

Luis Garcia received his PhD in 1989 at the University Paris 7 (Diderot) in the laboratory of Michel Fardeau (INSERM U153/UA614 CNRS) on the role of the L-type Ca2+ channel in excitation-contraction coupling in skeletal muscle. After a Post-doctoral experience (1989-1990) at the Rockefeller University in the Laboratory of Prof. Gerald M. Edelman, he was appointed by the CNRS (1991) as a permanent researcher. In 1995, Luis Garcia integrated the INSERM Unit «Neuroplasticity and Therapeutics»,

headed by Marc Peschanski (INSERM U421) to explore raising fields such as cell or gene therapy. In 1997, he joined Olivier Danos at the Genethon/UMR8115 CNRS laboratory to initiate its transformation into a Research Centre on gene therapy, and later on in 2007, Luis Garcia moved my team to the University Pierre & Marie Curie (INSERM U974/CNRS UMR7215) with the aim of developing therapeutic strategies based on mRNA repair. In 2012, he was invited to join the new Faculty of Medicine of the University of Versailles St-Quentin to create an Associated International Laboratory UVSQ-CSM in Monaco (LIA-BAHN - 2013) and a joint research laboratory UVSQ-INSERM in Versailles (U1179 - 2015), both committed to investigating novel therapeutic approaches for neuromuscular disabilities in relationship with the University hospitals of “Paris-Ile-de-France Ouest”. less

Science

Antisense Technology

Synthena AG is a development stage biotechnology company aiming to develop and commercialize new life saving drugs for the treatment of severe neuromuscular diseases based on the modulation of RNA. Its proprietary tricyclo-DNA technology platform offers broad advantages over state of the art oligonucleotide chemistries for new RNA intervention strategies like splice switching, and other antisense approaches. Its most advanced preclinical drug development program focuses on Duchenne Muscular Dystrophy, a genetic disorder which affects 1/3,500 boys and which leads to death in early adulthood.

Much as LNA, tricyclo(tc)-DNA has been designed as a conformationally constrained oligonucleotide analogue. Chemically, tc-DNA deviates from natural DNA by three additional C-atoms between C(5’) and C(3’). These chemical modifications change the properties of natural oligodeoxynucleotides in the following way:

Due to their properties tc-oligonucleotides are particularly suited for biological and therapeutic applications where high target affinity and biostability is required and where the mechanism of action does not rely on RNaseH activity.

Duchenne muscular dystrophy (DMD) is the consequence of a X-chromosome linked genetic defect, caused by a mutation in the dystrophin gene. Dystrophin is the largest known human gene consisting of 79 exons interrupted by 78 introns. Mutations can lead to exon skipping during splicing which in turn leads to misaligned mRNA that produces unfunctional protein. The incidence rate of the disease is ca 1 out of 3500 newborn males. The majority of mutations are spontaneous and not hereditary.

Dystrophin binds to the intracellular actin filaments and links via the trans-membrane sarcoglycans to the extracellular matrix. It is essential for muscle function.